Table 5.
Previous studies about target therapy on sarcoma
| Drug | Combined with chemotherapy | The first author’s last name | Year of publication | Trial Sponsor | Number of patients (N) | Clinical outcome |
|---|---|---|---|---|---|---|
| osteosarcoma | ||||||
| GT | Elizabeth Fox | 2012 | SARTCSf | 14 | ORR 7.1%; | |
| Sorafenib | no | Grignani | 2011 | Italian Sarcoma Group | 35 | 4 m-PFSa 46%; DRd 4 m; ORRc14%; |
| Trastuzumab | Cytotoxic Chemotherapy | Ebb | 2012 | COGe | 41 | 30 m-EFS 32%; 30 m-OSb 50%; without significant difference comparing with control group |
| Sirolimus | Cyclophosphamide | Schuetze | 2012 | Michigan University | 5 | ORR 0%; 4 m-PFS 30% (combined with other sarcoma) |
| Cixutumumab and Temsirolimus | no | Schwartz | 2013 | MSKCCg fund | 24 | ORR 13%; median PFS 6w |
| Cixutumumab | no | Weigel | 2014 | COG | 11 | ORR 0%; 1/11 SD for 140 d |
| R1507 | no | Pappo | 2014 | SARTCSf | 38 | ORR 2.5%; DR: 12w; 12w-PFS 17% |
| Sorafenib; Everolimus | no | Grignani | 2015 | Italian Sarcoma Group | 38 | 6 m-PFS 45%; DR 5 m; ORR 10% |
| Cixutumumab; Temsirolimus | no | Wagner | 2015 | COG | 11 | ORR 0%; |
| Dasatinib | no | Schuetze | 2016 | SARTCS | 46 | ORR 6.5%; DR 5.7 m; 2y-OS 15% |
| Apatinib | no | Present study | 2017 | 22 | ORR 40.9%;median PFS 3.1 m; 4 m PFS 24.1%; 6 m PFS 18.1% | |
| Ewing sarcoma | ||||||
| GT | Elizabeth Fox | 2012 | SARTCS | 14 | ORR 14.3%; | |
| R1507 | no | Pappo | 2011 | SARTCS | 115 | ORR 9.6%; median PFS 1.3 m; median OS 7.6 m |
| Figitumumab | no | Juergens | 2011 | European organization | 106 | ORR 14.2%; median PFS 1.9 m; median OS 8.9 m |
| Cixutumumab + temsirolimus | no | Schwartz | 2013 | MSKCC | 27 | ORR 14.8%; median PFS 7.5w; median OS 16.2 m |
| Olaparib | no | Choy E | 2014 | MGHh | 12 | ORR 0%; DR 5.7w; |
| Cixutumumab + temsirolimus | no | Wagner LM | 2015 | COG | 43 | ORR 0%; 12w-PFS 16%; |
| Apatinib+everolimus & apatinib alone | no | Present study | 2017 | 10 | ORR 70%; median PFS 2.0 m; 12w-PFS 22.5% | |
| Soft tissue sarcoma | ||||||
| Topotecan +carboplatin | Bochennek K | 2013 | CSTSGi | 34 | ORR 38%; | |
| Pazopanib | no | Winette T A | 2012 | EORTCj and the PALETTE study group | 246 | ORR 6%; median PFS 4.6 m; median OS 12.5 m |
| Olaratumab | Doxorubicin | William D Tap | 2016 | MSKCC and 16 clinical sites in US | 15 in IB trial and 67 in II trial | ORR 18.2%; median PFS 6.6 m; median OS 26.5 m |
| Regorafenib | no | Thomas Brodowicz | 2015 | International trial (France, Austria, Germany) | 82 | Median PFS 5.6 m for SS and 2.9 m for none specific; |
| Apatinib alone & apatinib+everolimus | Sometimes accompanied with GTk | Present study | 2017 | 21 | ORR 71.4%; median PFS 6.6 m; 4 m-PFS 71.4%; median OS 9.9 m | |
| chondrosarcoma | ||||||
| GT | Elizabeth Fox | 2012 | SARTCS | 25 | ORR 8%; | |
| GDC-0449 | no | A. Italiano | 2013 | French Sarcoma Group/US; French National Cancer Institute | 39 | ORR 0%; median PFS 3.5 m; 6-m PFS 28.2%; 1-y PFS 19.2% |
| Imatinib | no | Grignani | 2011 | Italian Sarcoma Group | 26 | ORR 0%; 4 m-PFS 31%; median OS 11 m; |
| Sirolimus | cyclophosphamide | Bernstein-Molho R | 2012 | Israel | 10 | ORR 10%; 60% SD for more than 6 m; median PFS 13.4 m |
| IDHl inhibitor | no |
NCT02273739; NCT02481154; NCT02073994; NCT02496741 |
2016–2017 | Under investigations | ||
| Apatinib alone | no | Present study | 2017 | 3 | ORR 100%; median PFS 7.37 | |
aPFS: progression-free survival
bOS: overall survival
cORR: overall response rate, defined as complete responses (CRs) + partial responses (PRs) + MRs;
dDR: Duration of response
eCOG: Children’s Oncology Group
fSARTCS: Sarcoma Alliance for Research Through Collaboration Study
gMSKCC: Memorial Sloan-Kettering Cancer Center
hMGH: Massachusetts General Hospital
iCSTSG: Cooperative Soft Tissue Sarcoma Group
jEORTC: European Organization for Research and Treatment of Cancer
kGT: chemo-protocol combined with gemcitabine 1000 mg/m2 d1,8 and docetaxel 75 mg/m2 d8
lIDH: isocitrate dehydrogenase